WEINHEIM, Germany, April 14, 2011 /PRNewswire/ -- International biotechnology firm Cytonet recently launched the first ever clinical trial in the United States using an investigational liver cell infusion to treat urea cycle disorders (UCD) in children. The first patient in the Phase II trial, which will seek to enroll 20 patients in 12 medical centers, is a child with citrullinemia, which is a hereditary UCD. He received six liver cell infusions on six consecutive days at Yale School of Medicine in New Haven, Conn.
UCDs are congenital and often life-threatening disorders of ammonia metabolism in the liver. Neurotoxic ammonia accumulates in the body and may lead – depending on the severity of the disease – to massive damage of the nerves and the brain and can be fatal. Children who remain untreated rarely experience normal physical and mental development. The only cure is liver transplantation, which is an extremely difficult procedure for very young children and neonatal patients. Additionally, there is a shortage of suitable organs available for transplantation.
"Liver cell infusion may provide a new treatment option for children," said Sukru H. Emre, MD, the lead investigator and Section Chief, Transplantation Surgery at Yale-New Haven Transplant Center. "We are looking forward to conducting this clinical trial."
The clinical trial, called SELICA III, is designed to evaluate the safety and efficacy of liver cell therapy in infants to children up to age 5 with UCD. The Investigational New Drug Application (IND) was submitted to the U.S. Food and Drug Administration (FDA) in mid-2010, following an analysis of interim results of an ongoing trial in Germany involving newborns with UCD. Cytonet received an Orphan Drug designation from the FDA's Office of Orphan Products Development.
Liver cell therapy involves collecting healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) which are then isolated and undergo complex processing. These cells are infused into the hepatic portal vein over six days. Risks include portal vein thrombosis and systemic shunting of liver cells.
"We are excited to help advance the landscape of UCD treatment through this milestone and are committed to continuing to identify patients who might benefit from this kind of therapy and find success," says Dr. Wolfgang Rudinger, CEO of Cytonet Germany.
For more information, please visit Cytonet's website at http://www.cytonetllc.com/
About the SELICA (Safety and Efficacy of Liver Cell Application) Study
For the past several years Cytonet has worked with internationally-leading metabolism centers to study its liver cell treatment. This therapy uses healthy and metabolically functional human hepatocytes for infusion to treat the metabolism disorder. In July 2010, the FDA permitted clinical testing to proceed in the SELICA III trial. The FDA was provided with an analysis of interim results of an ongoing trial (SELICA V) in Germany among newborns with UCDs. The SELICA III trial is being supported by the National Urea Cycle Disorders Foundation, a U.S. non-profit organization.
A urea cycle disorder is a genetic disorder caused by a deficiency of one of the six enzymes in the urea cycle which is responsible for removing ammonia from the blood stream. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to urea. Normally, the urea is transferred into the urine and removed from the body. In urea cycle disorders, the nitrogen accumulates in the form of ammonia, a highly toxic substance, and is not removed from the body resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it causes irreversible brain damage, coma and/or death.
Urea cycle disorders are included in the category of inborn errors of metabolism. Inborn errors of metabolism represent a substantial cause of brain damage and death among newborns and infants. Because many cases of urea cycle disorders remain undiagnosed and/or infants born with the disorders die without a definitive diagnosis, the exact incidence of these cases is unknown and underestimated. It is believed that up to 20 percent of Sudden Infant Death Syndrome cases may be attributed to an undiagnosed inborn error of metabolism such a urea cycle disorder. In April 2000, research experts at the Urea Cycle Consensus Conference estimated the incidence of the disorders at 1 in 10,000 births. This represents a significant increase in case identification and diagnosis in the last few years. Research studies have now been initiated to more accurately determine the incidence and prevalence of UCDs.
The Cytonet Group is an internationally active biotechnology company which is located in Weinheim and Heidelberg in Germany and in Durham, North Carolina in the U.S. The company develops, produces and markets cell therapeutic products. By using specially prepared human cells, it is possible to provide alternatives to existing therapies for many diseases. In addition, Cytonet provides blood stem cells and bone marrow preparations for the treatment of leukemia and other tumor diseases. The managing directors are Dr. Wolfgang Rudinger and Michael J. Deissner (MBA). Cytonet was founded as a result of the demerger of the cell therapy division from the firm of Roche in April 2000. The controlling interest is held by the family of Dietmar Hopp. The project is supported by the German Federal Ministry for Education and Research (BMBF) as part of the Leading Edge Cluster Competition (Spitzencluster Wettbewerb). Cytonet is an active partner in the Biotech-Cluster-Rhein-Neckar (BioRN) - the Rhine Neckar biotechnology cluster, which was designated in 2008 as the leading edge cluster in "cell-based and molecular medicine."
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